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DNL343 is an investigational CNS penetrant eukaryotic initiation factor 2B activator that prevents and reverses the effects of neurodegeneration caused by the integrated stress response Yulyaningsih, Ernie; Suh, Jung H.; Fanok, Melania; Chau, Roni; Solanoy, Hilda; Takahashi, Ryan; Bakardjiev, Anna, I; Becerra, Isabel; Benitez, N. Butch; Chiu, Chi-Lu; Davis, Sonnet S.; Dowdle, William E.; Earr, Timothy; Estrada, Anthony A.; Gill, Audrey; Ha, Connie; Haddick, Patrick C. G.; Henne, Kirk R.; Larhammar, Martin; Leung, Amy W-S; Maciuca, Romeo; Memarzadeh, Bahram; Nguyen, Hoang N.; Nugent, Alicia A.; Osipov, Maksim; Ran, Yingqing; Rebadulla, Kevin; Roche, Elysia; Sandman, Thomas; Wang, Jing; Lewcock, Joseph W.; Scearce-Levie, Kimberly; Kane, Lesley A.; Sanchez, Pascal E. 分享 收藏
Discovery of DNL343: A Potent, Selective, and Brain-Penetrant eIF2B Activator Designed for the Treatment of Neurodegenerative Diseases Craig II, Robert A.; De Vicente, Javier; Estrada, Anthony A.; Feng, Jianwen A.; Lexa, Katrina W.; Canet, Mark J.; Dowdle, William E.; Erickson, Rebecca I.; Flores, Brittany N.; Haddick, Patrick C. G.; Kane, Lesley A.; Lewcock, Joseph W.; Moerke, Nathan J.; Poda, Suresh B.; Sweeney, Zachary; Takahashi, Ryan H.; Tong, Vincent; Wang, Jing; Yulyaningsih, Ernie; Solanoy, Hilda; Scearce-Levie, Kimberly; Sanchez, Pascal E.; Tang, Liwei; Xu, Musheng; Zhang, Rui; Osipov, Maksim 分享 收藏
DNL310 normalizes primary storage substrates, corrects biomarkers of lysosomal dysfunction and reduces biomarkers of neuronal injury (neurofilament light chain) in MPS II: 2-year interim analysis of a phase 1/2 study Bhalla, Akhil; Tsogtbaatar, Khishig; Tran, Chau; Ravi, Ritesh; Fang, Meng; Zubizarreta, Imanol; Caprihan, Rupa; Karunakara, Ajith; Zhu, Yuda; Sinclair, Elizabeth; Jacquemet-Engelhart, Dominique; Kast, Johannes; Hung, Tony; Scearce-Levie, Kimberly; Troyer, Matt; Chin, Peter; Meirelles, Katia; Bakardjiev, Anna 分享 收藏
The Integrated Stress Response Is Modulated by eIF2B Agonist DNL343: Results From Phase 1 Healthy Subject and Phase 1b ALS Patient Studies Sun, Linus; Tsai, Richard; Yulyaningsih, Ernie; Fanok, Melania; Vissers, Maurits; Heuberger, Jules; Flores, Brittany; Huang, Fen; Kane, Lesley; Cohen, Isaac; Dhuria, Shyeilla; Fang, Meng; Estrada, Anthony; Osipov, Maksim; Willman-Yoswa, Brent; Maciuca, Romeo; Dobbins, Sarah; Chau, Ronnie; Earr, Timothy; Nguyen, Hoang; Lopez, Isabel; Scearce-Levie, Kimberly; Bunte, Tommy; Van den Berg, Leonard; Ho, Carole; Groeneveld, Geert-Jan; Troyer, Matthew 分享 收藏
LRRK2 Inhibition by BIIB122 in Healthy Participants and Patients with Parkinson's Disease Jennings, Danna; Huntwork-Rodriguez, Sarah; Vissers, Maurits F. J. M.; Daryani, Vinay M.; Diaz, Dolores; Goo, Marisa S.; Chen, John J.; Maciuca, Romeo; Fraser, Kyle; Mabrouk, Omar S.; van de Wetering de Rooij, Jeroen; Heuberger, Jules A. A. C.; Groeneveld, Geert Jan; Borin, Marie T.; Cruz-Herranz, Andres; Graham, Danielle; Scearce-Levie, Kimberly; De Vicente, Javier; Henry, Anastasia G.; Chin, Peter; Ho, Carole; Troyer, Matthew D. 分享 收藏
Discovery of Potent and Selective Dual Leucine Zipper Kinase/ Leucine Zipper-Bearing Kinase Inhibitors with Neuroprotective Properties in In Vitro and In Vivo Models of Amyotrophic Lateral Sclerosis Craig II, Robert A.; Fox, Brian M.; Hu, Cheng; Lexa, Katrina W.; Osipov, Maksim; Thottumkara, Arun P.; Larhammar, Martin; Miyamoto, Takashi; Rana, Anil; Kane, Lesley A.; Yulyaningsih, Ernie; Solanoy, Hilda; Nguyen, Hoang; Chau, Roni; Earr, Timothy; Kajiwara, Yuji; Fleck, Daniel; Lucas, Anthony; Haddick, Patrick C. G.; Takahashi, Ryan H.; Tong, Vincent; Wang, Jing; Canet, Mark J.; Poda, Suresh B.; Scearce-Levie, Kimberly; Srivastava, Ankita; Sweeney, Zachary K.; Xu, Musheng; Zhang, Rui; He, Jianrong; Lei, Yanan; Zhuo, Zheng; de Vicente, Javier 分享 收藏
Novel App knock-in mouse model shows key features of amyloid pathology and reveals profound metabolic dysregulation of microglia Xia, Dan; Lianoglou, Steve; Sandmann, Thomas; Calvert, Meredith; Suh, Jung H.; Thomsen, Elliot; Dugas, Jason; Pizzo, Michelle E.; DeVos, Sarah L.; Earr, Timothy K.; Lin, Chia-Ching; Davis, Sonnet; Ha, Connie; Leung, Amy Wing-Sze; Hoang Nguyen; Chau, Roni; Yulyaningsih, Ernie; Lopez, Isabel; Solanoy, Hilda; Masoud, Shababa T.; Liang, Chun-chi; Lin, Karin; Astarita, Giuseppe; Khoury, Nathalie; Zuchero, Joy Yu; Thorne, Robert G.; Shen, Kevin; Miller, Stephanie; Palop, Jorge J.; Garceau, Dylan; Sasner, Michael; Whitesell, Jennifer D.; Harris, Julie A.; Hummel, Selina; Gnorich, Johannes; Wind, Karin; Kunze, Lea; Zatcepin, Artem; Brendel, Matthias; Willem, Michael; Haass, Christian; Barnett, Daniel; Zimmer, Till S.; Orr, Anna G.; Scearce-Levie, Kimberly; Lewcock, Joseph W.; Di Paolo, Gilbert; Sanchez, Pascal E. 分享 收藏
Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson's disease Jennings, Danna; Huntwork-Rodriguez, Sarah; Henry, Anastasia G.; Sasaki, Jennifer C.; Meisner, Rene; Diaz, Dolores; Solanoy, Hilda; Wang, Xiang; Negrou, Elvira; Bondar, Vitaliy V.; Ghosh, Rajarshi; Maloney, Michael T.; Propson, Nicholas E.; Zhu, Yuda; Maciuca, Romeo D.; Harris, Laura; Kay, Angela; LeWitt, Peter; King, T. Alex; Kern, Drew; Ellenbogen, Aaron; Goodman, Ira; Siderowf, Andrew; Aldred, Jason; Omidvar, Omid; Masoud, Shababa T.; Davis, Sonnet S.; Arguello, Annie; Estrada, Anthony A.; de Vicente, Javier; Sweeney, Zachary K.; Astarita, Giuseppe; Borin, Marie T.; Wong, Bradley K.; Wong, Harvey; Nguyen, Hoang; Scearce-Levie, Kimberly; Ho, Carole; Troyer, Matthew D. 分享 收藏
Safety, pharmacokinetics and target engagement of novel RIPK1 inhibitor SAR443060 (DNL747) for neurodegenerative disorders: Randomized, placebo-controlled, double-blind phase I/Ib studies in healthy subjects and patients 新型RIPK1抑制剂SAR443060 (DNL747) 治疗神经退行性疾病的安全性，药代动力学和靶标参与: 健康受试者和患者的随机，安慰剂对照，双盲I/Ib期研究 Vissers, Maurits F. J. M.; Heuberger, Jules A. A. C.; Groeneveld, Geert Jan; Nijhuis, Jerome Oude; De Deyn, Peter Paul; Hadi, Salah; Harris, Jeffrey; Tsai, Richard M.; Cruz-Herranz, Andres; Huang, Fen; Tong, Vincent; Erickson, Rebecca; Zhu, Yuda; Scearce-Levie, Kimberly; Hsiao-Nakamoto, Jennifer; Tang, Xinyan; Chang, Megan; Fox, Brian M.; Pomponio, Robert J.; Alonso-Alonso, Miguel; Zilberstein, Moshe; Atassi, Nazem; Troyer, Matthew D.; Ho, Carole 分享 收藏
Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme 分子结构决定了转铁蛋白受体靶向溶酶体酶的脑递送 Arguello, Annie; Mahon, Cathal S.; Calvert, Meredith E. K.; Chan, Darren; Dugas, Jason C.; Pizzo, Michelle E.; Thomsen, Elliot R.; Chau, Roni; Damo, Lorna A.; Duque, Joseph; Fang, Meng; Giese, Tina; Kim, Do Jin; Liang, Nicholas; Nguyen, Hoang N.; Solanoy, Hilda; Tsogtbaatar, Buyankhishig; Ullman, Julie C.; Wang, Junhua; Dennis, Mark S.; Diaz, Dolores; Gunasekaran, Kannan; Henne, Kirk R.; Lewcock, Joseph W.; Sanchez, Pascal E.; Troyer, Matthew D.; Harris, Jeffrey M.; Scearce-Levie, Kimberly; Shan, Lu; Watts, Ryan J.; Thorne, Robert G.; Henry, Anastasia G.; Kariolis, Mihalis S. 分享 收藏
Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome Arguello, Annie; Meisner, Rene; Thomsen, Elliot R.; Nguyen, Hoang N.; Ravi, Ritesh; Simms, Jeffrey; Lo, Iris; Speckart, Jessica; Holtzman, Julia; Gill, Thomas M.; Chan, Darren; Cheng, Yuhsiang; Chiu, Chi-Lu; Dugas, Jason C.; Fang, Meng; Lopez, Isabel A.; Solanoy, Hilda; Tsogtbaatar, Buyankhishig; Zhu, Yuda; Bhalla, Akhil; Henne, Kirk R.; Henry, Anastasia G.; Delucchi, Anthony; Costanzo, Simona; Harris, Jeffrey M.; Diaz, Dolores; Scearce-Levie, Kimberly; Sanchez, Pascal E. 分享 收藏
Antibody semorinemab reduces tau pathology in a transgenic mouse model and engages tau in patients with Alzheimer's disease Ayalon, Gai; Lee, Seung-Hye; Adolfsson, Oskar; Foo-Atkins, Corinne; Atwal, Jasvinder K.; Blendstrup, Mira; Booler, Helen; Bravo, Joseph; Brendza, Robert; Brunstein, Flavia; Chan, Ruby; Chandra, Priya; Couch, Jessica A.; Datwani, Akash; Demeule, Barthelemy; DiCara, Danielle; Erickson, Rich; Ernst, James A.; Foreman, Oded; He, Dongping; Hotzel, Isidro; Keeley, Michael; Kwok, Michael C. M.; Lafrance-Vanasse, Julien; Lin, Han; Lu, Yanmei; Luk, Wilman; Manser, Paul; Muhs, Andreas; Ngu, Hai; Pfeifer, Andrea; Pihlgren, Maria; Rao, Gautham K.; Scearce-Levie, Kimberly; Schauer, Stephen P.; Smith, William B.; Solanoy, Hilda; Teng, Edmond; Wildsmith, Kristin R.; Yadav, Daniela Bumbaca; Ying, Yong; Fuji, Reina N.; Kerchner, Geoffrey A. 分享 收藏
Evaluation of fluid biomarkers reveals lysosome dysfunction and neurodegeneration in neuronopathic MPS II patients Bhalla, Akhil; Ravi, Ritesh; Fang, Meng; Arguello, Annie; Davis, Sonnet S.; Chiu, Chi-Lu; Blumenfeld, Jessica R.; Nguyen, Hoang N.; Earr, Timothy K.; Wang, Junhua; Astarita, Giuseppe; Zhu, Yuda; Fiore, Damian; Scearce-Levie, Kimberly; Diaz, Dolores; Cahan, Heather; Troyer, Matthew D.; Harris, Jeffrey M.; Escolar, Maria L. 分享 收藏
Iduronate-2-sulfatase transport vehicle rescues neurobehavioral and skeletal phenotypes in a mouse model of mucopolysaccharidosis type II Arguello, Annie; Meisner, Rene; Thomsen, Elliot R.; Ravi, Ritesh; Simms, Jeffrey; Speckart, Jessica; Holtzman, Julia; Gill, Michael; Cheng, Yuhsiang; Chiu, Chi-Lu; Fang, Meng; Nguyen, Hoang N.; Solanoy, Hilda; Tsogtbaatar, Buyankhishig; Zhu, Yuda; Bhalla, Akhil; Henry, Anastasia G.; Delucchi, Anthony; Costanzo, Simona; Harris, Jeffrey M.; Diaz, Dolores; Troyer, Matthew D.; Scearce-Levie, Kimberly; Sanchez, Pascal E. 分享 收藏
Longitudinal biomarkers in amyotrophic lateral sclerosis Huang, Fen; Zhu, Yuda; Hsiao-Nakamoto, Jennifer; Tang, Xinyan; Dugas, Jason C.; Moscovitch-Lopatin, Miriam; Glass, Jonathan D.; Brown, Robert H., Jr.; Ladha, Shafeeq S.; Lacomis, David; Harris, Jeffrey M.; Scearce-Levie, Kimberly; Ho, Carole; Bowser, Robert; Berry, James D. 分享 收藏
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GluN2A NMDA Receptor Enhancement Improves Brain Oscillations, Synchrony, and Cognitive Functions in Dravet Syndrome and Alzheimer's Disease Models Hanson, Jesse E.; Ma, Keran; Elstrott, Justin; Weber, Martin; Saillet, Sandrine; Khan, Abdullah S.; Simms, Jeffrey; Liu, Benjamin; Kim, Thomas A.; Yu, Gui-Qiu; Chen, Yelin; Wang, Tzu-Ming; Jiang, Zhiyu; Liederer, Bianca M.; Deshmukh, Gauri; Solanoy, Hilda; Chan, Connie; Sellers, Benjamin D.; Volgraf, Matthew; Schwarz, Jacob B.; Hackos, David H.; Weimer, Robby M.; Sheng, Morgan; Gill, T. Michael; Scearce-Levie, Kimberly; Palop, Jorge J. 分享 收藏
DNL104, a Centrally Penetrant RIPK1 Inhibitor, Inhibits RIP1 Kinase Phosphorylation in a Randomized Phase I Ascending Dose Study in Healthy Volunteers Grievink, Hendrika W.; Heuberger, Jules A. A. C.; Huang, Fen; Chaudhary, Rinkal; Birkhoff, Willem A. J.; Tonn, George R.; Mosesova, Sofia; Erickson, Rebecca; Moerland, Matthijs; Haddick, Patrick C. G.; Scearce-Levie, Kimberly; Ho, Carole; Groeneveld, Geert Jan 分享 收藏
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